Gene editing treats disease in mice

A team at Duke University used a system known as CRISPR-Cas9 to delete DNA that was preventing cells from producing a protein essential for muscle function. And a virus was used to deliver DNA alterations into the cells of mice.

Writing in the journal Science, the team say when they injected the therapy direct into the legs of adult mice, it resulted in improved muscle strength.

When they injected it into the bloodstream – tests showed improvements in muscles responsible for heart and lung function.
Chris Nelson, who led the research, said: “A major hurdle for gene editing is delivery.

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